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托伐普坦与生长抑素治疗 ADPKD:文献复习。

Tolvaptan vs. somatostatin in the treatment of ADPKD: A review of the literature.

出版信息

Clin Nephrol. 2022 Mar;97(3):131-140. doi: 10.5414/CN110510.

Abstract

Autosomal dominant polycystic kidney disease (ADPKD) is the most common genetic disorder with an estimated prevalence between 1 : 1,000 and 1 : 2,500. Until a few decades ago, ADPKD was considered an untreatable disease, relentlessly progressing towards end-stage renal disease because of the lack of specific interventions. In the last decade, some aberrant molecular pathways involved in ADPKD development have been identified, and controlled clinical trials have been conducted to investigate the potential role of active drugs on these pathophysiological mechanisms such somatostatin and tolvaptan. Somatostatin analogues have been shown to be effective not only in ADPKD, but also in polycystic liver disease (PLD) with beneficial effect on cardiac function and a better cost/benefit profile; the only somatostatin analogue currently available for clinical use is octreotide long-acting release (octreotide-LAR), and it is approved only in Italy. On the contrary, tolvaptan is authorized worldwide and has received more attention in the last years, even if its clinical use is widely limited by aquaresis tolerability. The aim of this review is to investigate the advantages and drawbacks of somatostatin analogues and tolvaptan in the treatment of ADPKD.

摘要

常染色体显性多囊肾病(ADPKD)是最常见的遗传性疾病之一,其发病率估计在 1:1000 至 1:2500 之间。直到几十年前,ADPKD 还被认为是一种无法治愈的疾病,由于缺乏特异性干预措施,它会无情地进展为终末期肾病。在过去的十年中,已经确定了一些涉及 ADPKD 发展的异常分子途径,并进行了对照临床试验,以研究活性药物对这些病理生理机制(如生长抑素和托伐普坦)的潜在作用。生长抑素类似物不仅在 ADPKD 中有效,而且在多囊肝病(PLD)中也有效,对心脏功能有有益影响,且具有更好的成本效益比;目前唯一可用于临床的生长抑素类似物是奥曲肽长效释放剂(octreotide-LAR),仅在意大利获得批准。相反,托伐普坦在全球范围内获得批准,并在过去几年中受到更多关注,尽管其临床应用受到耐水利尿的限制。本综述旨在探讨生长抑素类似物和托伐普坦在 ADPKD 治疗中的优缺点。

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